Epidermolysis Bullosa (RDEB)

Topical QR-313 in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Due to Mutation(s) in Exon 73 of the COL7A1gene (WINGS)

A First in Human, Double-blind, Randomized, Intra-subject Placebo-controlled, Multiple Dose Study of QR-313 Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With RDEB Due to Mutation(s) in Exon 73 of the COL7A1 Gene

Phase 1/2

DESCRIPTION:

Four medical centers in CA, CO, MN, and OH are recruiting participants who have specific types of Recessive Dystrophic Epidermolysis Bullosa to test a new, topical treatment. The treatment aims to slow progression of EB wounds through a drug that binds to and stops the cellular messaging system that causes inflammation.

The actual drug and a placebo will be applied 2 to 3x per week to two wound areas, as assigned by the study, for 4 weeks. Home and site visits will follow for regular assessments up to 8 weeks post the last dose.

 

PATIENT MUST:

  • Be 6 years of age or older
  • Have at least 1 target wound area
  • Have life expectancy > 6 months

THE STUDY INVOLVES:

  1. Screening before the treatment.
  2. Visit to the study center for assignment of the drug and/or placebo to the targeted wounds.
  3. For 4 weeks, the medicines will be applied to the assigned wounds 2 to 3 times per week.
  4. Home and site visits will follow for regular assessments up to 8 weeks post the last dose.

LOCATIONS AND CONTACTS:

The study locations include:

SPONSOR INFORMATION:

ProQR Therapeutics

Or go online: https://clinicaltrials.gov/ct2/show/NCT03605069